dystrophy — TargetMol Chemicals

Cat No.	Product Name	Synonyms	Targets
T39516	Bocidelpar		PPAR
	Bocidelpar is a modulator of PPARδ with an EC50 of 7.80 nM and improves mitochondrial biogenesis and function in Duchenne Muscular Dystrophy muscle cells.		PPAR
T7837	SR8278		Others
	SR8278 is an REV-ERBα antagonist(EC50 = 0.47 µM), blocking activation of the receptor by the synthetic agonist GSK 4112		Others
T11553	Heptamidine dimethanesulfonate	SBi4211 dimethanesulfonate	Others
	Heptamidine dimethanesulfonate (SBi4211 dimethanesulfonate) serves as a potent inhibitor related to Pentamidine, targeting the calcium-binding protein S100B with a dissociation constant (Kd) of 6.9 µM. It demonstrates sp...		Others
T11861	LM11A-31 dihydrochloride		Others
	LM11A-31 dihydrochloride is a water-soluble, non-peptide with high blood-brain barrier permeability.LM11A-31 dihydrochloride, a p75NTR (neurotrophin receptor p75) Ligand, is a potent proNGF (nerve growth factor) antagoni...		Others
T17217	Vamorolone	VBP15	Glucocorticoid Receptor , NF-κB
	Vamorolone (VBP15) is an orally active dissociative steroidal anti-inflammatory drug and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone displays effective NF-κB inhibition an...		Glucocorticoid Receptor , NF-κB
T3424	Ezutromid	BMN 195,VOX-C1100,SMT C1100	Others
	Ezutromid (BMN 195) is a translation modulator of Small utrophin (EC50: 0.4 uM). Ezutromid is an orally bioavailable small molecule that transcriptionally upregulates the utrophin gene, increasing both utrophin mRNA and ...		Others
T13491	Neuromuscular-targeting compound 1		Others
	Neuromuscular-targeting compound 1 can be used to study muscular dystrophy and neuromuscular-related diseases.		Others
T1805	Ataluren	PTC124	CFTR , Autophagy
	Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and olde...		CFTR , Autophagy
T9821	ART-CHEM-BB B025267		Others
	ART-CHEM-BB B025267 is the upregulator of utrophin production with EC50 of 1.8 μM and can be used in research on the treatment of Duchenne muscular dystrophy.		Others
T72072	2-benzylsulfanyl-6-methoxy-4-methylquinazoline		ROS
	2-benzylsulfanyl-6-methoxy-4-methylquinazoline can be used as a TRPML modulator for the treatment of diseases associated with TRPML activity, such as lysosomal storage disease, muscular dystrophy, oxidative stress or rea...		ROS
T72073	AKOS037652256
	AKOS037652256 can be used as a TRPML modulator for the treatment of diseases associated with TRPML activity such as lysosomal accumulation disorders, muscular dystrophy, common age-related neurodegenerative diseases, oxi...
T15945	MA-0204		PPAR
	MA-0204 is a highly selective and orally available peroxisome proliferator-activated receptor δ (PPARδ) modulator (EC50s: 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively). It is a potential treatmen...		PPAR
T21792	CGP 3466B maleate	Omigapil (Maleate),Omigapil maleate	Apoptosis
	CGP 3466B maleate (Omigapil maleate) is an orally bioavailable GAPDH nitrosylation inhibitor. Omigapil maleate abrogates Aβ1-42-induced tau acetylation, memory impairment, and locomotor dysfunction in mice. Omigapil male...		Apoptosis
T38554	Eteplirsen	AVI 4658
	Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research for Duchenne muscular dystrophy.
T38860	Golodirsen	Golodirsen,SRP-4053
	Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD).
T39517	Suvodirsen	WVE-210201
	Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD).
T66481	TG-693
	TG693, an orally available inhibitor of CLK1, promotes the skipping of the endogenous mutated exon 31 in Duchenne muscular dystrophy (DMD) patient-derived cells and increased the production of the functional exon 31-skip...
T34426	RTC14	Read-through compound-14,RTC 14,RTC-14,Read-through compound 14
	RTC14, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
T34425	RTC13	Read-through compound 13,RTC 13,RTC-13
	RTC13, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
T11553L	Heptamidine	SBi4211	Others
	Heptamidine is an effective Pentamidine-related inhibitor of the calcium-binding protein S100B (Kd: 6.9 μM). It selectively kills melanoma cells with S100B over those without S100B. It is a useful tool for the investigat...		Others